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Q2 2013 Conference Call July 25, 2013

Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

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Page 1: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Q2 2013 Conference CallJuly 25, 2013

Page 2: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Agenda

Patrick Flanigan, VP, Investor Relations

Bob Hugin, Chief Executive Officer

Jackie Fouse, Chief Financial Officer

Mark Alles, Head of Hematology/Oncology

Q&A

Scott Smith, Head of Inflammation & Immunology

2

Q&A

Page 3: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Forward Looking Statements and Adjusted Financial Information

This presentation contains forward-looking statements, which are generally statements that are nothistorical facts. Forward-looking statements can be identified by the words “expects,” “anticipates,”“believes,” “intends,” “estimates,” “plans,” “will,” “outlook” and similar expressions. Forward-looking, , , p , , p gstatements are based on management’s current plans, estimates, assumptions and projections, andspeak only as of the date they are made. We undertake no obligation to update any forward-lookingstatement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict andare generally beyond our control Actual results or outcomes may differ materially from those impliedare generally beyond our control. Actual results or outcomes may differ materially from those impliedby the forward-looking statements as a result of the impact of a number of factors, many of whichare discussed in more detail in our Annual Report on Form 10-K and our other reports filed with theSecurities and Exchange Commission.

In addition to financial information prepared in accordance with U.S. GAAP, this presentation alsocontains adjusted financial measures that we believe provide investors and management withsupplemental information relating to operating performance and trends that facilitate comparisonsbetween periods and with respect to projected information. These adjusted financial measures arenon-GAAP and should be considered in addition to but not as a substitute for the informationnon GAAP and should be considered in addition to, but not as a substitute for, the informationprepared in accordance with U.S. GAAP. We typically exclude certain GAAP items thatmanagement does not believe affect our basic operations and that do not meet the GAAP definitionof unusual or non-recurring items. Other companies may define these measures in different ways.Further information relevant to the interpretation of adjusted financial measures, and reconciliations

f th dj t d fi i l t th t bl GAAP b f d

3

of these adjusted financial measures to the most comparable GAAP measures, may be found onCelgene’s website at www.Celgene.com in the “Investor Relations” section.

Page 4: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Bob Hugin

Page 5: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Q2 2013: A Quarter of Outstanding Results

Exceptional Financial and Commercial Results:Strong Y/Y growth: net product sales ↑17% revenue ↑17% adjusted diluted EPS ↑25%Strong Y/Y growth: net product sales ↑17%, revenue ↑17%, adjusted diluted EPS ↑25%REVLIMID® continued expansion and positive momentumSuccessful POMALYST® launch in the US and ABRAXANE® trajectory

Advancing the Portfolio:Approvals for REVLIMID® in MCL in US and del5q MDS in EU Positive CHMP opinion for pomalidomidePositive CHMP opinion for pomalidomideStatistically significant PFS in MM-020 trialApremilast PsA submission in US; PSOR and EU submissions on track for H2

Building for the Future:Strengthening the early pipeline organically and through collaborationsDiscovery platforms producing development candidates

5

Page 6: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Advancing Towards the Next Phase of Growth

Maximize Full Potential of our Hematology Franchise1 Maximize Full Potential of our Hematology Franchise1

Expand our Oncology Franchise into New Indications2

Submit Apremilast with Regulators and Prepare for Launch3

Advance Early-Stage Clinical Programs to Key Decision Points4

6

Page 7: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Jackie Fouse

Page 8: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Q2 2013 Financial Highlights

Excellent operating results across the P&L:Year-over-year net product sales grew 17% and adjusted diluted EPS grew 25%Year over year net product sales grew 17% and adjusted diluted EPS grew 25%Improved adjusted operating margins by 180 bps Y/Y; 20 bps Q/Q

Adding value with financial drivers:Adding value with financial drivers:Authorized additional $3 billion in share repurchase programTotal repurchases in Q2 $834M, 6.9M shares; H1:13 $1.84B, 16.3M shares

Strong momentum in metrics including:REVLIMID® year-over-year growth of 13% U S l f REVLIMID® f MCL EU l f MDSU.S. approval for REVLIMID® for MCL; EU approval for MDS

Investing for the future:Entered into additional collaborations addressing new drug targetsExecuting on investment plan for I&I

8

Page 9: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Total Net Product Sales

Q2(Growth Rates = Growth vs. Prior Year Period)

$1,337

$1,564

$1,148*

$ ,

on

↑40% ↑16%$ M

illio

↑17%

Q2:11 Q2:12 Q2:13*Q2:11 adjusted

9

Page 10: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Volume Drove Q2:13 Net Product Sales Growth

Contribution to Q2:13 Net Product Sales Growth(Growth Rates = Growth vs. Prior Year Period)

$1 600

$1,800 ↑17%↓2%↑16% ↑3%

$1,200

$1,400

$1,600

on

$600

$800

$1,000

$ M

illio

$200

$400

$600

$0Q2:12 Volume Price Fx / Hedge Q2:13

10

Page 11: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Adjusted Diluted Earnings Per Share

Q2(Growth Rates = Growth vs. Prior Year Period)

$1.52

$0.89

$1.22

r sha

re

↑37%↑29% ↑25%

olla

rs p

erD

Q2:11 Q2:12 Q2:13

11

Page 12: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Adjusted Diluted EPS Growth Driven by Operating Income

Contribution to Q2:13 Adjusted Diluted EPS

$1.52$0.00$0.26$1.22 ($0.01) $0.05

Sha

reol

lars

Per

D

Q2:12 Operating Income Financial Income / Expense

Tax Rate Share Count Q2:13

12

Page 13: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Worldwide Net Product Sales

Net Product Sales ($ million)

Q22013

∆ vs.Q2:12

∆ vs.Q1:13

REVLIMID® Total $1,052 ↑13% ↑5%

U.S. $625 ↑16% ↑10%

International $427 ↑8% ↓2%

VIDAZA® Total $211 ↑5% ↑4%U.S. $84 ↑3% ↓3%

International $127 ↑7% ↑8%International $127 ↑7% ↑8%

ABRAXANE® Total $155 ↑41% ↑26%

U.S. $120 ↑38% ↑28%

International $35 ↑55% ↑21%

POMALYST® Total $66 NA NA

U S $58 NA NAU.S. $58 NA NA

International $8 NA NA

13

Page 14: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Volume Drove Q2:13 REVLIMID®

Net Product Sales Growth

Contribution to Q2:13 REVLIMID® Net Product Sales Growth(Growth Rates = Growth vs. Prior Year Period)

$1,200 ↑12.6%↓2.5%↑11.4% ↑3.7%

$800

$1,000

on

$600

$800

$ M

illio

$200

$400

$0Q2:12 Volume Price Fx / Hedge Q2:13

14

Page 15: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Key P&L Line Items (Adjusted)

Q2 ∆ vs. ∆ vs.Q2013 Q2:12 Q1:13

Product Gross Margins 95.1% ↑30 bps ↑50 bpsProduct Gross Margins 95.1% ↑30 bps ↑50 bps

R&D Expenses% of revenue

$345M21.5% ↓400 bps ↓100 bps

SG&A Expenses% of revenue

$384M24.0% ↑240 bps ↑120 bps

Operating Profit Margin 49.6% ↑180 bps ↑20 bps

Effective Tax Rate 17.1% ↑60 bps ↑60 bps

15

Page 16: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Adjusted Operating Leverage Increased

37.9%41.0%

44.3% 45.1%48.1% 49.5%

28.0%

25.4% 23.4% 22 8% 22.8% 22.0%24.1%

26.0% 25.5% 25.8%23.8% 23.4%

25.4% 23.4% 22.8% 22.8% 22.0%10.6% 8.0% 7.1% 6.5% 5.4% 5.2%

2008 2009 2010 2011 2012 YTD 2013

Operating Margin SG&A R&D COGS

16

Page 17: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Improving ROIC

16%$12

ROIC

12%

14%$10

8%

10%

$6

$8

on

4%

6%$4$

Bill

io

0%

2%

$0

$2 Average Invested Capital

0%$02007 2008 2009 2010 2011 2012 2013

(TTM)

17

GAAP operating income used for all periods except 2008. Refer to reconciliation tables for ROIC calculation methodology

Page 18: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Cash and Marketable Securities

($ billion) 6/30/13 12/31/12($ )

Cash and Marketable Securities $4.08 $3.90

• Cash flow from operations was $737M during Q2• In Q2 2013, purchased 6.9M shares for total cost

$834M • In H1 2013 purchased 16 3M shares for total costIn H1 2013, purchased 16.3M shares for total cost

$1.84B• Authorized additional $3 billion in share repurchase

program

18

Page 19: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

2013 Adjusted Guidance Raised

2013 Guidance

∆ vs.2012 Previous

Total Net Product Sales ~$6.2B ↑~15% ~$6B

REVLIMID® Net Product $4 2 4 3B ↑~13% $4 1 4 2BSales $4.2-4.3B ↑~13% $4.1-4.2B

Adjusted Diluted EPS $5.80-$5 90 ↑~19% $5.55-

$5 65

• Key Assumptions

• Share count remains constant with YE:12

j $5.90 ↑ $5.65

Share count remains constant with YE:12

• Adjusted Operating Margins of ~49%

• Range accommodates a possible generic VIDAZA® entry in Q4:13

U i id i t f t l l t th• Using midpoint of range to calculate growth

• Includes investment to build I&I franchise

19

Page 20: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Mark Alles

Page 21: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Q2 Hematology/Oncology Operating Results

Record Net Product Sales:Year-over-year net product sales of $1,564M grew 17% Year-over-year REVLIMID® sales of $1 052M grew 13%Year-over-year REVLIMID sales of $1,052M grew 13%Year-over-year ABRAXANE® sales of $155M grew 41%POMALYST® sales of $66M; H1 2013 sales of $95M

Product Growth Drivers Intact:New clinical data, publications, presence at international meetingsNew indication and product approvals commercial launchesNew indication and product approvals, commercial launchesMarket share and duration of therapy gains, geographic expansion

Significant Franchise Expansion Opportunities:REVLIMID® global approvals for NDMM, novel combinations in multiple myelomaPOMALYST® global approvals for RRMM

21

REVLIMID® CLL and NHL development programsGlobal approvals for ABRAXANE® in pancreatic cancer

Page 22: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Q2 2013 REVLIMID® Results

EuropeUnited States Rest-of-World• Y/Y sales grew 9%

• Strong performance in Germany, Italy, and UK

• EU4 multiple myeloma

• Y/Y sales grew 16%

• Overall multiple myeloma market share consistently in mid 50% range

• Good quarterly sales in Japan, Canada and Australia

• China launch underway; • EU4 multiple myeloma market share (2nd line) consistently above 50% and growing

• Strong duration of

g

• Total prescriptions increased; record number of patients treated

• Increased duration of

y;early results exceed internal expectations

• Achieved public reimbursement in • Strong duration of

therapy gains in France and Italy

• Launching recently approved MDS

• Increased duration of therapy

• Launching RR MCL indication

Mexico; seeking reimbursement in Korea, Russia, and Brazil

approved MDS indication

22

Page 23: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Planned Expansion of REVLIMID® in NDMM: MM-020 Design and Objectives

Trial ArmsPatients

Trial Arms

N = 1,623 N l di d

ARM A: REVLIMID® / low dose dexamethasone continuous therapy

to disease progression• Newly diagnosed

• Transplant-ineligiblemultiple myeloma

to disease progression

ARM B:REVLIMID® / low dose dexamethasone for 18 28-day cycles

R1:1:1

• Stratified by age(65-75 vs. > 75 years), disease stage (ISS I/II vs. III) and country

ARM C: THALOMID® / melphalan / prednisone for 12 42-day cyclescountry prednisone for 12 42-day cycles

• Primary Endpoint: Progression-free survival• Secondary Endpoint: Overall survival

23

Page 24: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Planned Expansion of REVLIMID® in NDMM: MM-020 Design and Objectives

Trial ArmsPatients

Trial Arms

N = 1623 N l di d

ARM A: REVLIMID® / low dose dexamethasone continuous therapy

to disease progressionEnrollment began August 2008• Newly diagnosed

• Transplant-ineligiblemultiple myeloma

to disease progression

ARM B:REVLIMID® / low dose dexamethasone for 18 28-day cycles

R1:1:1

Enrollment began August 2008Enrollment completed in Q1 2011

Achieved Primary Endpoint – Final PFS• Stratified by age(65 - 75 vs. > 75 years) and disease stage (ISS I/II vs. III) ARM C: THALOMID® / melphalan /

prednisone for 12 42-day cycles

Achieved Primary Endpoint Final PFS

prednisone for 12 42-day cycles

• Primary Endpoint: Progression-free survival• Secondary Endpoint: Overall survival

24

Page 25: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

REVLIMID® Newly Diagnosed Multiple Myeloma:Phase III Data and Regulatory Update

Trial Relevant PatientP l ti

Possible Timing f M t D t

Possible RegulatoryTrial Population of Mature Data Regulatory Submission Dates

MM-020 NDMM NSCT Elderly

Q3:13(Final PFS)

Q4:13 EUQ4:13 USElderly (Final PFS)

(Interim OS)Q4:13 US

MM-015 NDMM NSCTMaintenance

Q1:13(OS Trend)

Q4:13 EU

< 75 years( )

IFM 2005-02 NDMM SCT Maintenance

H2:14/H1:15(OS Trend)

6 months after availability of data

CALGB 100104

NDMM SCT Maintenance

H2:14/H1:15(OS Trend)

6 months after availability of data

25

Page 26: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

REVLIMID® Maintenance Improved PFS and OS in NDMM (GIMEMA)

N=402

Source: Palumbo, et. al, ASCO 2013 and EHA 2013

26

Page 27: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Key REVLIMID® Lymphoma Data from the International Congress on Malignant Lymphoma (ICML) Meeting

CALGB 50803 N=63

• Phase II study of REVLIMID® Plus rituximab in patients with

Dose 20-25 mg

Duration 1 year

rituximab in patients with previously untreated follicular lymphoma (CALGB 50803)

Rituximab 8 doses

Median Age 53 years

FLIPI 0-1 32%

• Multicenter phase II demonstrating R2 produced 93% overall response rate (ORR), >70% PET

FLIPI 2 65%

FLIPI 3-5 3%

ORR 93%

response rate (ORR), 70% PET (–) CR

• Previous phase II (Fowler et al) ORR 93%

CR 72%

PFS Too early

• Previous phase II (Fowler, et al) demonstrated >90% ORR with very high CR rates

Source: Martin, et.al., ICML 2013

27

Page 28: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

RELEVANCE® Trial of REVLIMID® plus Rituximab®

in Untreated Follicular Lymphoma

Trial ArmsPatients

Trial Arms

REVLIMID® : 20-mg on D2-22 of a 2-day cycle for up to 18 cycles

Rituximab: 375 mg/m2 D 1 8 15 and 22 of aN = 1,000

R1:1

Rituximab: 375 mg/m2 D 1, 8, 15 and 22 of a 28-day cycle for cycles 2-6, then 375 mg/m2

every 8 weeks for 12 cycles• Histologically confirmed

CD20+ follicular lymphoma grade 1, 2 or 3a

N l di d

Rituximab-CHOP, Rituximab-CVP, Rituximab-Bendamustine followed by 375 mg/m2 Rituximab every 8 weeks for 12 cycles

• Newly diagnosed

• Stage II, III or IV disease

28

• Primary Endpoint: Complete response rate; Progression free survival• Secondary Endpoints: Safety, time to treatment failure, event free survival, time to next anti-

lymphoma treatment, time to next chemotherapy treatment, overall survival, QoL

Page 29: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

RELEVANCE® Trial of REVLIMID® plus Rituximab®

in Untreated Follicular Lymphoma

Trial ArmsPatients

Trial Arms

REVLIMID® : 20-mg on D2-22 of a 2-day cycle for up to 18 cycles

Rituximab: 375 mg/m2 D 1 8 15 and 22 of aN = 1,000

R1:1

Rituximab: 375 mg/m2 D 1, 8, 15 and 22 of a 28-day cycle for cycles 2-6, then 375 mg/m2

every 8 weeks for 12 cycles• Histologically confirmed

CD20+ follicular lymphoma grade 1, 2 or 3a

N l di d

Enrollment On Target>35% of Patients Enrolled

Rituximab-CHOP, Rituximab-CVP, Rituximab-Bendamustine followed by 375 mg/m2 Rituximab every 8 weeks for 12 cycles

• Newly diagnosed

• Stage II, III or IV disease Accrual Expected to Complete H2:14

29

• Primary Endpoint: Complete response rate; Progression free survival• Secondary Endpoints: Safety, time to treatment failure, event free survival, time to next anti-

lymphoma treatment, time to next chemotherapy treatment, overall survival, QoL

Page 30: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

REMARC® REVLIMID® Maintenance in Diffuse Large B-Cell Lymphoma (DLBCL)

Trial ArmsPatients

Trial Arms

REVLIMID®

N = 621

R1:1

Daily for D1-21 of a 28-day cycleN = 621

• Patients who have received at least 6-8 cycles of the R-CHOP 14 or R-CHOP 21

Placebo

• Stratification by high risk

30

• Primary Endpoint: Progression free survival• Secondary Endpoints: Overall survival, event-free survival, response rate, percentage of patients

who convert from PR (partial response) to CR (complete response), adverse events

Page 31: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

REMARC® REVLIMID® Maintenance in Diffuse Large B-Cell Lymphoma (DLBCL)

Trial ArmsPatients

Trial Arms

REVLIMID®

N = 621

R1:1

Daily for D1-21 of a 28-day cycleN = 621

• Patients who have received at least 6-8 cycles of the R-CHOP 14 or R-CHOP 21

Enrollment On Target~75% of Patients Enrolled

Placebo

• Stratification by high riskAccrual expected to complete in Q4:13

31

• Primary Endpoint: Progression free survival• Secondary Endpoints: Overall survival, event-free survival, response rate, percentage of patients

who convert from PR (partial response) to CR (complete response), adverse events

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CLL-002 CONTINUUM® Trial of REVLIMID®

Maintenance in Chronic Lymphocytic Leukemia (CLL)

Trial ArmsPatients

Trial Arms

REVLIMID® N = 400

• Treated with a purine

R1:1

Daily until disease progressionp

analog- or bendamustine-containing regimen in the 1st and/or 2nd line induction therapy

Placebo

• Alemtuzumab-containing regimens allowed for those patients with 17p deletion

32

• Primary Endpoint: Overall survival, progression free survival• Secondary Endpoints: Safety, tumor response, duration of response, health related quality of life

Page 33: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

CLL-002 CONTINUUM® Trial of REVLIMID®

Maintenance in Chronic Lymphocytic Leukemia (CLL)

Trial ArmsPatients

Trial Arms

REVLIMID® N = 400

• Treated with a purine

R1:1

Daily until disease progressionp

analog- or bendamustine-containing regimen in the 1st and/or 2nd line induction therapy

Enrollment On Target; >60% EnrolledAccrual expected to complete in H2:14

Placebo

• Alemtuzumab-containing regimens allowed for those patients with 17p deletion

Accrual expected to complete in H2:14

33

• Primary Endpoint: Overall survival, progression free survival• Secondary Endpoints: Safety, tumor response, duration of response, health related quality of life

Page 34: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

POMALYST®

U.S. Launch Update

• Strong launch momentum continues– Registered prescribers

N ti t t t i ti– New patient starts, prescriptions– Repeat prescriptions– Positive payer and market access awareness– Executing multiple myeloma franchise commercial strategy

• Positive physician and patient clinical experience

• Data presentations at ASCO and EHA updated clinical profile– Long-term OS follow up from MM-002, MM-003, and IFM 0902– Quality of life results– Data in patients with renal Impairment– Data in patients with renal Impairment– Cytogenetically defined high risk disease– Elderly patients– Novel combinations

34

Page 35: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Q2 2013 ABRAXANE® Results

• Q2:13 Net Sales: $155M, ↑41% Y/Y– US ↑38% Y/Y, International ↑55% Y/Y

• Breast Cancer$155

Breast Cancer– US market share stable; gains in

Germany, Italy and Spain– Initiating Triple Negative Breast Cancer

study$106 $106

$123

study

• NSCLC– Continued US market share gains in

overall and squamous histologyI iti ti i t t d i lio

ns

Int'lUS

– Initiating maintenance study in squamous NSCLC

• Pancreatic Cancer– World GI Symposium presentations

$ M

ill– Advancing adjuvant phase III study– PDUFA date September 21, 2013

• Other Tumor Types Q3:12 Q4:12 Q1:13 Q2:13– Melanoma phase III study expect mature

overall survival results by Q4:13

35

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Generating Strong Franchise Momentum

Our Offer significant value propositions OurProducts • Improved clinical outcomes

• Excellent global market access

OurPerformance

Focused on operational excellence• 2013 net product sales of approximately $6.2B

N li i l d t i di ti d d t l• New clinical data, new indication and product approvals

OurOutlook

Long growth runway with multiple drivers• Meaningful catalysts for growth in multiple myeloma,

MDS, NHL, CLL, and oncology

36

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Scott Smith

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Rapid Succession of Approvals Expected Through 2014 and Beyond

S b it

On Track

S b it I l t

Submit Australia and Switzerland PSOR/PsA

S b it

Submit EU MAA AS

PDUFA DateMarch 21

Submit EU Approval

Submit Canada

PSOR NDS

Approval Approval Approval

Implement next tier

registrations

Submit US AS NDA

Submit CanadaAS NDS

Approval US AS

March 21, 2014

Submit US PsA NDA

Submit EU PsA/PSOR

MAA

Submit USPSOR NDA

Approval US PsA

Approval US PSOR

Submit Canada

PsA NDS

Approval Canada

PsA

Approval CanadaPSOR

ApprovalEU

PsA/PSOR

Q1Q1 Q2Q2 H1H1 H2H2 H1H1H2H2

2013 2014E 2015E2013 2014E 2015E

38

• Exploring opportunities to file Behçet’s

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PALACE Program: Consistent Response Rate that Improves over Time

ACR 20 t W k 16ACR 20 t W k 16 PALACE 1 52 WeeksPALACE 1 52 Weeks

60

70

43 §

PALACE 1 PALACE 3

ACR 20 at Week 16ACR 20 at Week 16

60

70

80

PALACE 1 52 WeeksPALACE 1 52 Weeks

g %)

Apremilast 20 mg BID Apremilast 30 mg BID

20

30

40

50Subjects Achieving

ACR20(%)

19

31*

41§

24

29*

43 §

30

40

50

Patie

nts

Ach

ievi

ngC

R20

Res

pons

e (%

* §P 0 05 P 0 0001

0

10

165 163 161 164 163 159n=

Placebo Apremilast 20 mg BID Apremilast 30 mg BID

0

10

20

16 24 40 52

P AC

Study Week Data as observed* §P>0.05; P>0.0001. LOCF, Per Protocol Populationy Data as observed

Source: Edwards, et.al., EULAR 2013 Source: Kavanaugh, et.al., EULAR 2013

39

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BCT 001: Signficant Oral Ulcer Response

Mean Number of Oral UlcersMean Number of Oral Ulcers

3 5

Oral Ulcer Response at Day 85Oral Ulcer Response at Day 85

2 0

2.5

3.0

3.5

70.9*

89.1*

80

100

(%)

Mean # of Oral Ulcers

0 5

1.0

1.5

2.0

28.6

50

40

60(%)

Patients Achieving Complete or Partial Oral Ulcer Response

wk0

wk2

wk4

wk6

wk8

wk10

wk12

wk14

wk16

wk18

wk20

wk22

wk24

wk26

wk28

Placebo 2.9 1.7 1.9 1.9 1.6 1.4 2.1 0.4 0.4 0.6 0.5 0.3 0.4 1.3 1.630 mg BID 2.7 0.3 0.7 0.5 0.5 0.7 0.5 0.6 0.6 0.6 0.7 0.2 0.6 1.9 1.7

0.0

0.5

0

20

Complete Response Partial Response

Placebo Apremilast 30 mg BID*P<0.0001Placebo Apremilast 30 mg BID

*P<0.0001

Source: Hatemi, et.al., EULAR 2013

40

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Robust Data Rollout at Medical Meetings

Data Release AnticipatedData Release AnticipatedppH2 2013H2 2013

PALACE Program (1-3)• PALACE 2 full data• PALACE 3 52-week update• Enthesitis/dactylitis data• Pooled metabolic/lab data

Behçet’s Disease Phase II DetailsESTEEM 1• Difficult-to-treat patient subsets

( l d il )(scalp and nails)• Patient reported outcomes• Safety data

41

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I&I: Transformational Potential for Patients and for Celgene

Apremilast• Positive efficacy data in two psoriasis and four

psoriatic arthritis trials

Potential to createa new gold standard

for treatment of

• Differentiated safety profile and well tolerated across studied patient populations

• Unique benefit risk profile for treatment of

psoriatic disease as well as other serious

immunologicdiseases

• Emerging profile supports significant opportunity across multiple segments of the market

• Lifecycle management underway diseasesLifecycle management underway

• Submissions on track

• Key management positions filled

• Launch activities accelerating• Launch activities accelerating

Robust pipeline under development

42

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Bob Hugin

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Key Milestones – 2013

Product Milestone Expected Timing

Phase III MM-020 data in NDMM Q2:13 / Q3:13

US decision for mantle cell lymphoma Q2:13US decision for mantle cell lymphoma Q2:13

• Resubmission of the NDMM application in EU H2:13

• Submit for approval in the US in NDMM H2:13

• Approvals and reimbursement for RRMM in emerging markets Throughout 2013

US Regulatory decision for RRMM Q1:13

• EU Regulatory decision for RRMM Q3:13

× Phase III myelofibrosis data H1:13

Submit regulatory applications for pancreatic cancer in US & EU H1:13

• Mature phase III overall survival data in melanoma H2:13

• FDA decision for pancreatic cancer Q3:13

Phase III ESTEEM data in psoriasis Q1:13Phase III ESTEEM data in psoriasis Q1:13

• Submit for approval in psoriatic arthritis and psoriasis US; ROW Throughout 2013

Phase III data in treatment-naïve psoriatic arthritis H1:13

• Complete enrollment in Phase III ankylosing spondylitis trial H2:13

44

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Q2 2013 Conference CallJuly 25, 2013

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Reconciliation Tables

Page 47: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Reconciliation Tables

Net product salesOther revenue

Total revenue

Cost of goods sold (excl uacquired intangible a

Research and developmeSelling, general and adminAmortization of acquired Acquisition related charg

Total costs and exp e

Operating income

Other income (expense),

Income before income ta

Income tax provision

Net income

Net income per common BasicDiluted

Weighted average shares

BasicDiluted

Balance sheet items:Cash, cash equivaleTotal assetsShort-term borrowi nLong-term debtTotal stockholders' e uding amortization of

assets)entnistrative

intangible assetses and restructuring, netenses

net

axesshare:

s:nts & marketable securities

ngsequity

CelgCondens

20

$

$

$ $

Ju n20

s$ 1

gene Corporation ansed Consolidated Sta

(Unaudited)(In millions, except per

Thre0132012

1,564.1

1,336.6$

34.9

30.2

1,599.01,366.8

80.9

71.9

458.1

447.2

418.1

323.0

65.7

44.1

12.5

39.3

1,035.3925.5

563.7

441.3

(5.9)

(0.6)

557.8

440.7

79.7

73.3

478.1

367.4$

1.15

0.84$

1.11

0.82$

414.1

436.7

429.3

445.4

ne 30,December 31,

0132012

4,081.4

3,900.3$

11,963.711,734.3

887.8

308.5

2,730.4

2,771.3

5,407.15,694.5

nd Subsidiariesatements of Income

share data)

ee-Month Periods EndedJune 30,

20132,993.4$

$ 70.2

3,063.6

161.4

910.5

787.1

131.4

45.7

2,036.1

1,027.5

(21.3)

1,006.2

143.2

863.0$

$

2.07$

$ 2.00

$ $

416.0

431.0

Six-Month Peri oJune 30

47

20122,582.1

58.0

2,640.1 144.4 809.2 648.8 85.9 28.2

1,716.5 923.6 (8.9) 914.7 145.8 768.9 1.76 1.72 437.5 447.1

ods Ended0,

Page 48: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Reconciliation Tables

Net income - GAAP

Before tax adjustm Cost of goods so of acquired inta Products exite Share-based c

Research and de Share-based c IPR&D impai Upfront collab

Selling, general a Share-based c

Amortization of a

Acquisition relate Change in fair Acquisition an

Net income tax adNet income - Adjusted

Net income per commoBasicDiluted

In addition to financiameasures that we belietrends that facilitate cand should be considetypically exclude certadefinition of unusual ments:

old (excluding amortizationangible assets):ed or to be exited -Pharmiocompensation expense

evelopment:compensation expenseirmentsboration payments

and administrative:compensation expense

acquired intangible assets

ed charges and restructurinr value of contingent considnd restructuring costs

djustmentsdon share - Adjusted

CeReconc

al information prepared ieve provide investors and

comparisons between periered in addition to, but nain GAAP items that manaor non-recurring items. O

$

on(1)

(2)

(2)

(3)

(4)

(2)

(5)

ng, net:deration

(6)

(6)

(7) $

$ $ T

elgene Corporationciliation of GAAP to

(In millions, except p

in accordance with U.S. Gd management with suppliods and with respect to pot as a substitute for, theagement does not believeOther companies may def 2013

2012

478.1

367$

-

(0

3.7

3

31.8

23

-

-

81.8

75

34.3

27

65.7

44

12.5

38

-

1

(55.0)

(34

652.9

54 4$

1.58

1.$

1.52

1 .$

Three-Month Periods Ended

n and Subsidiarieso Adjusted Net Incoper share data)

June 30,

GAAP, this press release lemental information relaprojected information. Th

e information prepared ine affect our basic operatiofine these measures in diff

2013

7.4863.0

$

0.6)-

3.0

6.5

3.658.8

-

-

5.0177.5

7.170.1

4.1131.4

8.145.7

1.2

-

4.3)(108.4)

4.6

1,244.6$

.252.99

$ .22

2.89$

dSix-Month P

ome

Jun

also contains adjusted fiting to operating perform

hese adjusted measures an accordance with U.S. GAons and that do not meet

fferent ways.

48

2012768.9$ (2.0) 5.9 48.6 22.2 75.0 53.9 85.9 25.6 2.6 (57.6) 1,029.0$

2.35$ 2.30$

Periods Endedne 30,

inancial mance and are non-GAAP

GAAP. We the GAAP

Page 49: Q2 2013 Conference Call · Q2 ∆ vs. ∆ vs. 2013 Q2:12 Q1:13 Product Gross MarginsProduct Gross Margins 95.1% ↑30 bps30 bps ↑50 bps50 bps R&D Expenses % of revenue $345M 21.5%

Reconciliation Tables

Explanation of adjustments:(1) Exclude the net (benefit) cost of activities arising from the acquisition of Pharmion Corp. (Pharmion) that are planned to be exited.(2) Exclude share-based compensation expense totaling $69.8 for the three-month period ended June 30, 2013 and $53.7 for the

three-month period ended June 30, 2012. Exclude share-based compensation expense totaling $135.4 for the six-month period endedJ 30 2013 d $108 4 f th i th i d d d J 30 2012 June 30, 2013 and $108.4 for the six-month period ended June 30, 2012.

(3) Exclude in-process research and development impairments recorded as a result of changes in estimated probability-weighted cash flows.(4) Exclude upfront payments for research and development collaboration arrangements.(5) Exclude amortization of intangible assets acquired in the acquisitions of Pharmion, Gloucester Pharmaceuticals, Inc. (Gloucester),

Abraxis BioScience Inc. (Abraxis) and Celgene Avilomics Research, Inc. (formerly known as Avila Therapeutics)(Avila).(6) Exclude acquisition related charges and restructuring including changes in the fair value of contingent consideration related to the(6) Exclude acquisition related charges and restructuring, including changes in the fair value of contingent consideration, related to the

acquisitions of Gloucester, Abraxis and Avila.(7) Net income tax adjustments reflect the estimated tax effect of the above adjustments and the impact of certain other non-operating

tax adjustments, including one-time effects of acquisition related matters, adjustments to the amount of unrecognized tax benefits and deferred taxes on unremitted foreign earnings.

49

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Reconciliation Tables

Projected net income -

Before tax adjust Cost of goods so of acquired int Share-based

Research and d Share-based Upfront coll a

Selling, general Share-based

Amortization of

Acquisition rela t Change in fai

Net income tax a

Projected net income -

Projected net income p

Projected net income p

Projected weighted av

(1)Our projected ear intangible asset occur after the d

Reconciliation o- GAAP

tments:old (excluding amortizationtangible assets):compensation expense

development:compensation expense

aboration payments

and administrative:compensation expense

facquired intangible assets

ted charges and restructurir value of contingent cons

adjustments

- Adjusted

per diluted common share

per diluted common share

verage diluted shares

rnings do not include the eimpairments, or changes i

date of this press release.

Celgene Corporof Full-Year 2013 P

(In millions

(1)$

n

s

ring, net:sideration

$

- GAAP$

- Adjusted$

effect of any business comin the fair value of our CV

ration and SubsidiaProjected GAAP to, except per share data)

LowHig

1,792.8$

1$

12.6

123.2

267.5

163.9

265.4

63.0

(194.4)

2,494.0$

2$

4.17$

$

5.80$

$

430.0

mbinations, collaboration aVRs issued as part of the a

Range

arieso Adjusted Net Incgh1,855.0

12.2 118.4 267.5 157.5 262.8 63.0 (199.4) 2,537.0

4.31 5.90 430.0 greements, asset acquisit iacquisition of Abraxis that

come

50

ions, t may

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Return on Invested Capital Calculation

Return on Invested Capital (ROIC)Return on Invested Capital (ROIC)2013 (TTM) 2012 2011 2010 2009 2008

Operating income 1,850,332 1,746,442 1,442,753 989,635 841,526 (1,464,218) Certain charges (1) 2,043,069

Operating income (non-GAAP for 2008) 1,850,332 1,746,442 1,442,753 989,635 841,526 578,851

Effective tax rate 13% 13% 7% 13% 20% 24%Effective tax rate 13% 13% 7% 13% 20% 24%Operating income after tax (non-GAAP for 2008) 1,617,883 1,512,428 1,339,017 860,221 669,930 439,272

Total equity 5,407,102 5,694,467 5,512,727 5,995,472 4,394,606 3,491,328 Certain charges (1) 1,979,510 1,979,510 1,979,510 1,979,510 1,979,510 1,979,510 Total debt 3,618,161 3,079,792 1,802,269 1,247,584 - -

Total capital 11,004,773 10,753,769 9,294,506 9,222,566 6,374,116 5,470,838Total capital 11,004,773 10,753,769 9,294,506 9,222,566 6,374,116 5,470,838

Total capital beginning of period 9,600,085 9,294,506 9,222,566 6,374,116 5,470,838 3,040,499 Total capital end of period 11,004,773 10,753,769 9,294,506 9,222,566 6,374,116 5,470,838

Average total capital 10,302,429 10,024,138 9,258,536 7,798,341 5,922,477 4,255,669

ROIC 15.7% 15.1% 14.5% 11.0% 11.31% 10.3%O C 5 % 5 % 5% 0% 3 % 0 3%

(1) Excludes $1.7 billion of IPR&D expense in 2008 associated with the acquisition of Pharmion, as well as $300 millionof expense related to the acquisition of intellectual property rights for Vidaza in 2008 prior to it's launch. Amounts adjustedfor tax effects in 2008 are excluded from equity in all years including and subsequent to 2008.

51

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Appendix

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Celgene Pipeline

53

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Celgene Pipeline

54

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Celgene Pipeline

55

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REVLIMID® Multiple Myeloma Late Stage Programs

Patient Population NDMM Non-ASCT Eligible NDMM Non-ASCT Eligible

Trial Name MM-015MM-020FIRST®

Phase III III

Target Enrollment 459 1,623

D i

Arm A: REVLIMID® (10mg), melphalan, prednisone for 9 cycles followed by :

REVLIMID® (10mg) maintenance to disease progression

Arm B: : REVLIMID® (10mg) melphalan

Arm A: : REVLIMID®/low-dose dexamethasone until disease progression

Arm B: : REVLIMID®/low-dose d th f 18 4 k lDesign Arm B: : REVLIMID (10mg), melphalan,

prednisone for 9 cycles followed by placebo maintenance to disease progression

Arm C: Melphalan/prednisone for 9 cycles followed by placebo maintenance to disease

progression

dexamethasone for 18 4-week cyclesArm C:

THALOMID®/melphalan/prednisone for 12 6-week cycles

Primary Endpoint Progression Free Survival Progression Free Survival

Study met primary endpoint July 2009Data presented at ASH 2009 with follow-up d t t ASCO 2010 ASH d IMW 2011

Enrollment completeStatus data at ASCO 2010, ASH and IMW 2011,

ASH 2012 and IMW 2013. Published in NEJM May 2012

Follow-up continuing

Trial met primary endpoint for PFSData presentation expected at ASH 2013

56

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REVLIMID® Multiple Myeloma Late Stage Programs

Patient Population Maintenance Post-ASCT Maintenance Post-ASCT

Trial Name CALGB 100104 IFM 2005-02Trial Name CALGB 100104 IFM 2005-02

Phase III III

Target Enrollment 459 614

Arm A: Single agent : REVLIMID® (10mg)Arm A: : REVLIMID®consolidation (25mg) for 2 cycles followed by REVLIMID® (10-

15mg) until disease progressionDesign until disease progression

Arm B: Placebo until disease progression

15mg) until disease progressionArm B: : REVLIMID®consolidation (25mg)

for 2 cycles followed by placebo until disease progression

P i E d i t Ti t P i P i F S i lPrimary Endpoint Time to Progression Progression Free Survival

Status

Trial met primary endpoint in Dec 2009Data presented at ASCO 2010. Follow-up data at ASH 2010, IMW 2011 and IMW

2013

Trial met primary endpoint in June 2010Data presented at ASCO 2010. Follow-up data at ASH 2010 and IMW 2011.Status 2013.

Published in NEJM May 2012.Follow-up for survival continuing

pPublished in NEJM May 2012.

Follow-up for survival continuing

57

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POMALYST® Multiple Myeloma Late Stage Programs

Patient Population RRMM (3rd Line +) RRMM

Trial NameMM-003 MM-007

Trial NameNIMBUS OPTIMISMM

Phase III III

Target Enrollment 426 782

Arm A: POMALYST® (4mg) and low- Arm A: POMALYST® (4mg), bortezomib (1.3

Design

Arm A: POMALYST (4mg) and lowdose dexamethasone to disease

progressionArm B: High-dose dexamethasone to

disease progression

Arm A: POMALYST (4mg), bortezomib (1.3 mg/m2 IV) and low-dose dexamethasone to

disease progressionArm B: Bortezomib (1.3 mg/m2 IV) and low-dose dexamethasone to disease progression

Primary Endpoint Progression Free SurvivalProgression Free Survival

Primary endpoint met Dec 2012Status

Primary endpoint met Dec 2012Data presented at ASH 2012 and ASCO

2013Trial enrolling

58

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POMALYST® Multiple Myeloma Late Stage Programs

Patient Population RRMM

T i l N MM-010Trial Name MM 010STRATUS

Phase IIIb

Target Enrollment 507

DesignPOMALYST® (4mg), bortezomib (1.3

mg/m2 IV) and low-dose dexamethasone to disease progression

Primary Endpoint Adverse Events

Status Trial enrolling

59

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MDS/AML/MF Late Stage Programs

Patient Population Non-del5Q low risk/INT-1 transfusion-dependent MDS

Low risk/INT-1 transfusion-dependent MDS

CC-486Molecule REVLIMID® (Oral Azacitidine)

Trial Name MDS-005 AZA-MDS-003

Phase III III

Target Enrollment 228 386

DesignArm A: REVLIMID® (10mg)

Arm B: PlaceboArm A: CC-486 (150- or 200-mg)

Arm B: Placebo

Primary Endpoint RBC-transfusion independencefor at least 8 weeks

RBC-transfusion independence formore than 12 weeks

Status Trial enrolling Trial enrolling

60

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MDS/AML/MF Late Stage Programs

Patient Population Elderly Newly Diagnosed AML Post induction AML Maintenance

MoleculeVIDAZA®

(azacitidine)CC-486

(oral azacitidine)(azacitidine) (oral azacitidine)

Trial Name AZA-AML-001 CC-486-AML-001

Phase III III

Target Enrollment 480 460Target Enrollment 480 460

Arm A: VIDAZA®

(75 mg/m2 SC) daily for D1-7 28-day cycleuntil disease progression

A B C ti l C R i t A A CC 486 (150 200 )Design

Arm B: Conventional Care Regimen to disease progression

Intensive chemotherapy Low-dose cytarabineBest Supportive Care

Arm A: CC-486 (150- or 200-mg)Arm B: Best Supportive Care

Best Supportive Care

Primary Endpoint Overall Survival Overall Survival

StatusEnrollment complete

Trial not open to enrollmentStatusTop-line data in early 2014(E)

Trial not open to enrollment

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MDS/AML/MF Late Stage Programs

Patient Population

Myeloproliferative-Neoplasm-Associated

Myelofibrosis

Molecule POMALYST®Molecule POMALYST®

Trial Name RESUME (MF-002)

Phase III

Target Enrollment 210

DesignArm A: POMALYST® 0.5mg

dailyArm B: Placebo

Primary Endpoint

Proportion of subjects achieving RBC-transfusion-

independence

Trial did not meet primary

StatusTrial did not meet primary

endpointData to be submitted to a future

meeting

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REVLIMID® Chronic Lymphocytic Leukemia Late Stage Programs

Patient Population Elderly Newly Diagnosed CLL Maintenance in 2nd Line CLL

Trial NameCLL-008 CLL-002

Trial NameORIGIN® CONTINUUM®

Phase III III

Target Enrollment 428 400

Arm A: REVLIMID® (starting dosage A A REVLIMID® ( t ti d

Design

(sta t g dosage5mg/day escalated to 10mg/day) until

disease progression. 28-day cycleArm B: Chlorambucil (0.8 mg/kg) D1-15 of 28-day cycle for ~13 cycles (12 months)

Arm A: REVLIMID® (starting dosage 2.5mg/day escalated to 10mg/day) until

disease progression. 28-day cycleArm B: Placebo

Primary Endpoint Progression Free Survival Overall Survival and Progression-Free Survival

Enrollment completed in Q1:13Status

Enrollment completed in Q1:13Trial put on clinical hold in July 2013

Data being analyzed

Trial enrollingEnrollment complete in H2:14(E)

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REVLIMID® Lymphoma Late Stage Programs

Patient PopulationMaintenance in Patients with

DLBCL responding to R-CHOP to induction therapy

Newly Diagnosed Follicular Lymphoma

Trial Name REMARC RELEVANCE®

Phase III III

Target Enrollment 621 1,000

Arm A: REVLIMID® (starting dose 20m) D2 22 of 28 da c cle for p to 18 c cles

DesignArm A: REVLIMID® D1-21 of 28-day

cycle for 24 monthsArm B: Placebo D1-21 of 28-day

cycle for 24 months

D2-22 of 28-day cycle for up to 18 cycles and rituximab (starting dose 375 mg/m2

weekly) for up to 12 cyclesArm B: Physician’s choice of rituximab-

CHOP, rituximab-CVP or rituximab-bendamustinebendamustine

Primary Endpoint Progression Free Survival Complete response rate and Progression Free Survival

64

StatusTrial enrolling

Enrollment complete in Q4:13(E)Trial enrolling

Enrollment complete in H2:14(E)

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ABRAXANE® Solid Tumor Late Stage Programs

Patient Population Metastatic Malignant Melanoma Metastatic Adenocarcinoma of the Pancreas

Trial Name CA033MPACT

Trial Name CA033CA046

Phase III III

Target Enrollment 514 842

Arm A: ABRAXANE® (150 mg/m2) D 1, 8,Arm A: ABRAXANE® (125 mg/m2) and gemcitabine (1000 mg/m2) weekly for 3

Design

Arm A: ABRAXANE (150 mg/m2) D 1, 8, and 15 of a 28-day cycle

Arm B: Cacarbazine (1000 mg/m2) D 1 with steroid and antiemetic of a 21-day

cycle

gemcitabine (1000 mg/m2) weekly for 3 weeks of a 4 week cycle

Arm B: Gemcitabine (1000 mg/m2) weekly for 7 weeks of an 8 week cycle(Cycle 1); Weekly for 3 weeks of a 4

week cycles (Cycle 2+)

Primary Endpoint Progression Free Survival Overall Survival

Efficacy/safety data presented at Society f M l

Primary endpoint met

65

Statusof Melanoma

Research Meeting in November 2012Mature OS data expected in 2013

Efficacy/safety data presented at ASCO GI in January 2013

Regulatory submissions in US and EU

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ABRAXANE® Solid Tumor Late Stage Programs

Patient Population First-Line Triple Negative Metastatic Breast Cancer

Trial NametnAcity™

Trial NameABI-007-MBC-001

Phase II/III

Target Enrollment 240-350

Design

Phase IIArm A: ABRAXANE® 125mg/m2,/Gemcitabine 1000 mg/m2, d 1 and 8 – 21-day cycle

Arm B: ABRAXANE® 125mg/m2/Carboplatin AUC 2 IV, d 1 and 8 – 21-day cycleArm C: Gemcitabine 1000 mg/m2/Carboplatin AUC 2 IV, d 1 and 8 – 21-day cycleg g y y

Phase IIIArm 1: Selected phase II ABRAXANE® arm

Arm 2: Gemcitabine 1000 mg/m2/Carboplatin AUC 2 IV, d 1 and 8 – 21-day cycle

Primary Endpoint Progression Free Survival

Status Enrolling Soon

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I&I Late Stage Programs

Patient Population

Moderate-to-SevereLate Stage Psoriatic

ArthritisModerate-to-Severe Late Stage Psoriatic Arthritis

Moderate-to-Severe Late Stage Psoriatic Arthritis

with Skin Lesions

M l l A il t A il t A il tMolecule Apremilast Apremilast Apremilast

Trial NamePALACE-1PSA-002

PALACE-2PSA-003

PALACE-3PSA-004

Phase III III III

Target Enrollment 495 495 495

Design

Arm A: Apremilast (20mg)twice daily

Arm B: Apremilast (30mg) twice daily

Arm C: Placebo

Arm A: Apremilast (20mg)twice daily

Arm B: Apremilast (30mg) twice daily

Arm C: Placebo

Arm A: Apremilast (20mg)twice daily

Arm B: Apremilast (30mg)twice daily

Arm C: Placebo

Primary Endpoint ACR20 ACR20 ACR20

Enrollment completeEffi / f t d t

Enrollment completeEnrollment complete

StatusEfficacy/safety data presented at ACR in

November 2012 and EULAR 2013

Top-line data in Sept 2012Efficacy/safety to be presented in 2013

Enrollment completeEfficacy/safety to bepresented in 2013

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I&I Late Stage Programs

Patient Population

Treatment Naïve Moderate-to-SevereLate Stage Psoriatic

Arthritis

Moderate-to-SeverePlaque Psoriasis

Moderate-to-Severe Plaque Psoriasis

Molecule Apremilast Apremilast Apremilast

Trial NamePALACE-4PSA-005

ESTEEM-1PSOR-008

ESTEEM-2PSOR-009

Phase III III III

Target Enrollment 495 825 825

Design

Arm A: Apremilast (20mg)twice daily

Arm B: Apremilast (30mg) twice daily

A C Pl b

Arm A: Apremilast (30mg) twice daily

Arm B: Placebo

Arm A: Apremilast (30mg)twice daily

Arm B: PlaceboArm C: Placebo

Primary Endpoint ACR20 PASI-75 PASI-75

Enrollment complete Enrollment complete Enrollment completeStatus

Enrollment completeEfficacy/safety to be presented in 2013

Enrollment completeEfficacy/safety data presented

at AAD in March 2013

Enrollment completeEfficacy/safety data to be

presented in 2013

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I&I Late Stage Programs

Patient Population Moderate-to-Severe Plaque Psoriasis Ankylosing Spondylitis

Molecule Apremilast ApremilastMolecule Apremilast Apremilast

Trial Name PSOR-010POSTURE

AS-001

Ph IIIb IIIPhase IIIb III

Target Enrollment 240 456

Design

Arm A: Apremilast (30 mg) twice dailyArm B: Etanercept (50 mg subcutaneous)

once weeklyArm C: Placebo

Arm A: Apremilast (20mg) twice dailyArm B: Apremilast (30mg) twice daily

Arm C: Placebo

Primary Endpoint PASI75 ASAS20

Trial enrollingStatus Trial enrolling

Trial enrollingEnrollment complete in H2:13(E)

Top line data expected in 2014

69